NorthWest Biotherapeutics Inc (NWBO)

[Dan88]

IMHO, definitely positive despite somebody may spin negatively otherwise. My simple interpretation of the quote from Bill Malloy interview with LL below is 1) it's impossible to design a trial without giving chance to patients the real shots after their GBMs progress [it's true in the past and it's true today. FDA has to adapt to it because the sponsors designing and running the trial have no way to change this situation;] 2)FDA has to catch up with the nature of immunotherapy for accepting appropriate measures for its efficacy. As a result, today we know FDA has exactly caught up and come up with this guidance:

Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products Guidance for Industry (https://www.fda.gov/media/133660/download)

And specifically, NWBIO has received Regulatory Agencies' acceptance of NWBIO's adapted reordered endpoints (https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-001977-13/GB, and https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-001977-13/DE)

"Q: Can you explain more about the difficulties in designing the trials to do what’s best for the patient?

A: For instance, I’ll just take what happened with the DCVax. You really want to get clean data. We’ve never allowed for patients to get the vaccine once they failed, because they will. You enrolled in the trial. You had a recurrence, and now you’ll have to try something else.

I could tell you patients are fighting to get the vaccine. And even if you tell them, “Well, there’s no proof that this works. This is the whole reason we have to do the trial. I can’t tell you that there’s any benefit.” But I guess the counterargument that I hear quite often is, “well, the side-effects aren’t bad.” And they’re not. So, it’s like, “Well, if it’s not going to hurt me why can’t I have it? Why can’t I try it?”

Because of that it’s hard to prove something based on what the FDA requirements are without being a little cold-hearted and saying, “Well, no, you’re not allowed to try that.”

So, a part of it is having the FDA think a little bit differently about what constitutes approval. On the converse, like the Optune device, that Novocure device, is FDA-approved. But I must say a lot of patients are like, “Well, I don’t really like it.” And I think on the physician side, we just don’t understand how it works and there are a lot of problems with the clinical trial design.

There was no true placebo arm, but they did design it in a way that met the FDA checkboxes. So, it was approved, but a lot of us don’t really believe that it works. I think it’s one of these things where if your ultimate goal is to cure GBM, it’s hard to reconcile all these different factors; the regulatory factors, the financial factors, as well as the science and the biology of the tumor itself, which is very complex.
Q: Got it. Sounds like we have our work cut out for us.

A: Yeah, but if your smart people can figure this out, I think it could be done. It’s been done in other fields, but I think it just takes a different way of thinking about it, and it’s not the “Eureka!” moment of, “oh, I discovered something in the lab. I’m going to take it to clinical trial, launch my startup company, and charge ABM.”"